I wonder who's going to be first in line to pay a premium for a drug that will (probably) expire (multiple times) before it's used, and isn't any more effective than the stuff they've already got?
It's much easier to get drugs approved via the orphan drug route. Once its approved, doctors can use it off-label. If they can convince some docs to use it off-label or show efficacy with an animal model...it's a much more valuable drug to them as it treats a condition that is way more prevalent that has only symptomatic management at this point.